A Study to Find How Safe and Effective GAMMAPLEX® is in Subjects With Chronic Idiopathic Thrombocytopenic Purpura (ITP)

A Phase III, Multicenter, Open-Label Study To Evaluate the Efficacy and Safety of GAMMAPLEX® in Chronic Idiopathic Thrombocytopenic Purpura

To determine if GAMMAPLEX raises the platelet count of subjects with chronic ITP to a threshold of 50 x 109/L, similar to that of published response >60%. Also to assess the safety of GAMMAPLEX and determine if platelet counts are maintained at 50 x 109/L in subjects with chronic ITP for.

Study Overview

• Status: Completed
• Conditions: Chronic Idiopathic Thrombocytopenic Purpura
• Intervention / Treatment: Biological: Gammaplex, intravenous immunoglobulin

Detailed Description

The primary objective is to determine if BPL's GAMMAPLEX raises the platelet count of subjects with chronic ITP to a threshold of 50 x 109/L, similar to that of published response >60%. The secondary objectives are: 1) to determine the safety of GAMMAPLEX at the dosage used in this study. 2) to determine if GAMMAPLEX maintains platelet counts of ³ 50 x 109/L in subjects with chronic ITP for a period of time similar to that of a published data.

• Study Type: Interventional
• Enrollment (Actual): 35
• Phase: Phase 3

Contacts and Locations

Study Locations

• Argentina
  • Buenos Aires, Argentina, C1280AEB
    • Hospital Britanico
  • Buenos Aires, Argentina, C1122AAL
    • I. A. D. T. (Instituto Argentino de Diagnóstico y Tratamiento)
  • Buenos Aires, Argentina, C1431FWO
    • Centro de Educación Médica e Investigaciones Clinicas Dr. Norberto Quirno (CEMIC)
  • Cordoba, Argentina, X5016KEH
    • Hospital Privado de Cordoba
  • Corrientes, Argentina
    • J.R. Vidal Hospital
  • San Juan, Argentina, 5400
    • Cer San Juan
  • Buenos Aires
    • Capital Federal, Buenos Aires, Argentina, C1437JCP
      • Hospital Churruca
    • La Plata, Buenos Aires, Argentina, B1900AXU
      • Hospital Italiano de La Plata
  • Sante Fe
    • Rosario, Sante Fe, Argentina, S2000JKR
      • Instituto de Diagnóstico Hematológico Ambulatorio (IDHEA)
• India
  • New Delhi, India, 110060
    • Sir Ganga Ram Hospital
  • Andhra Pradesh
    • Hyderabad, Andhra Pradesh, India, 500003
      • Apollo Hospitals
    • Hyderabad, Andhra Pradesh, India, 500054
      • Mahavir Hospital
    • Suryarao pet, Vijayawada, Andhra Pradesh, India, 520002
      • City Cancer Centre,
  • Bangalore
    • Karnataka, Bangalore, India, 560054
      • M. S Ramaiah Hospital
  • Gujarat
    • Ahmedabad, Gujarat, India, 380009
      • Vedanta
    • Ahmedabad, Gujarat, India, 380061
      • Gurukrupa Hospital
    • Gokal Nagar, Karamsad, Gujarat, India, 388325
      • M S Patel Cancer Centre, Shree Krishna Hospital
  • Karnataka
    • Bangalore, Karnataka, India, 560017
      • Manipal Hospital
    • Mangalore, Karnataka, India, 575002
      • Father Muller Medical College Hospital
    • Mangalore, Karnataka, India, 5750003
      • Vinaya Hospital & research Centre
    • Mangalore, Karnataka, India, 575001
      • Kasturba Medical College, Manipal Acunova KMC Research Centre
  • Maharashtra
    • Pune, Maharashtra, India, 411004
      • Deenanath Mangeshkar Hospital
  • Rajasthan
    • Jaipur, Rajasthan, India, 300201
      • S.K. Soni Hospital Sect 5
• United States
  • Florida
    • Orange City, Florida, United States, 32763
      • Mid Florida Hematology & Oncology
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Rush University Medical Center
  • Maryland
    • Bethesda, Maryland, United States, 20817
      • Center for Cancer & Blood Disorders
  • New York
    • New York, New York, United States, 10021
      • New York Hospital / Cornell University, Division of Pediatrics
    • Stony Brook, New York, United States, 11794-8111
      • Department of Pediatrics, SUNY at Stony Brook
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health & Science University
  • Texas
    • San Antonio, Texas, United States, 78229
      • Cancer Care Centers of South Texas

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Males and females aged between 18 and 70 years.
2. Confirmed diagnosis of chronic ITP of at least 6 months duration.
3. Platelet count of less than or equal to 20 x 10 9/L at enrollment.
4. Absence of other conditions that, in the opinion of the investigator, could cause thrombocytopenia.
5. If subjects were treated with corticosteroids the treatment regimen/dose must have been stable (for a minimum of 2 weeks before screening). The dose of corticosteriod or other immunosuppressant should have remained constant until Day 32. 6) If subjects were being treated with cyclophosphamide, azathioprine or attenuated androgens, the treatment regimen and dose must have been stable for a minimum of 2 months before Day 1. The dose of corticosteriod or other immunosuppressant should have remained constant until Day 32. 7) Splenectomized subjects and both Rh(D)+ and Rh(D)- subjects may be included.

8) The subject has signed an informed consent form (subjects must be at least 18 years old), and/or the subject's legal guardian has signed the informed consent form if indicated 9) If a subject is a female of child-bearing potential, she must have a negative result on a urine-based HCG pregnancy test.

10) If a subject is a female who is or becomes sexually active, she must practice contraception by using a method of proven reliability for the duration of the study.

Exclusion Criteria:

1. A history of any severe or anaphylactic reaction to blood or any blood-derived product, or any severe reaction to IGIV or any other IgG preparation.
2. Intolerance to any component of the investigational product.
3. Received any live virus vaccine within the last 3 months prior to Day1.
4. Received an IGIV preparation within 1 month prior to Day 1.
5. Were currently receiving, or has received, any investigational agent within the 1 month prior to Day 1.
6. Received any blood, blood product, or blood derivative within the 1 month prior to Day 1.
7. Received Rituximab within the 3 months before Day 1.
8. Pregnant or nursing.
9. Tested positive for any of the following at screening: HBsAg, NAT for HCV, NAT for HIV, Antibodies to HCV or HIV 1 or 2.
10. Had levels greater than 2.5 times the upper limit of normal at screening, as defined by the central laboratory, of alanine aminotransferase or aspartate aminotransferase.
11. Had severe renal impairment (defined as serum creatinine greater than 2 times the upper limit of normal or BUN greater than 2.5 times the upper limit of normal for the range of the laboratory doing the analysis); on dialysis; a history of acute renal failure.
12. Known to have abused alcohol, opiates, psychotropic agents, or other chemicals or drugs within the past 12 months.
13. History of deep vein thrombosis (DVT) or thrombotic complications of IGIV therapy.
14. Any history or sign of hyperviscosity, transient ischemic attack (TIA), stroke, other thromboembolic event, or unstable angina.
15. Suffered from any acute or chronic medical conditions (e.g., renal disease or predisposing conditions for renal disease, coronary artery disease, or protein losing enteropathy) that, in the opinion of the investigator, may interfere with the conduct of the study.
16. An acquired medical condition, such as chronic lymphocytic leukemia, lymphoma, multiple myeloma, chronic or recurrent neutropenia (defined as an absolute neutrophil count (ANC) < 1 x 109/L).
17. Non-controlled arterial hypertension (systolic blood pressure >160 mmHg and/or diastolic blood pressure >90 mmHg).
18. Anemic (hemoglobin <10 g/dL) at screening.
19. Unlikely to adhere to the protocol requirements of the study or is likely to be uncooperative.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Gammaplex (intravenous immunoglobulin)

Biological: Gammaplex, intravenous immunoglobulin; Dosage form: Gammaplex® is a sterile liquid of 5 % w/v normal immunoglobulin. Gammaplex® contains 5 g/100 mL of human normal immunoglobulin (i.e. 50 g/L, of which virtually 100% is IgG). The first course of GAMMAPLEX will be administered as an intravenous infusion of 1 g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen may be administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX.; Other Names: Gammaplex

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Number of Subjects With Chronic ITP Treated With Gammaplex Whose Platelet Count Reached a Threshold of 50 x 10^9/L.	The number of subjects with chronic ITP treated following treatment with Gammaplex who attained a platelet count of ≥ 50 x 10^9/L by Day 9.	9 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Safety of GAMMAPLEX at the Dosage Used in This Study.	The safety variables used to assess safety were the following: Adverse eventsThe number and percent of infusions with at least 1 adverse event(AE) that occurs during an infusion or within 72 hours after the infusion stopsNature, severity, and frequency of AEsSuspected unexpected serious adverse reactions (SUSARs); Vital signs; Clinical laboratory tests and Direct Coombs' Test; Transmission of viruses; Physical examination	AEs were documented from the date the informed consent form was signed until the End of Study visit on Day 90.
Duration of Time That the Platelet Count of Subjects With Chronic ITP Treated With Gammaplex Remained ≥ 50 x 10^9/L.	Blood samples were collected to measure platelet counts and the duration of time for which the platelet count remained ≥50 x 10^9/L was measured.	Days 1, 2, 3, 5, 9, 14, 21, 32.

Collaborators and Investigators

• Sponsor: Bio Products Laboratory
• Investigators: Principal Investigator: Tim J Aldwinckle, MD, Bio Products Laboratory

Publications and helpful links

General Publications

• Dash CH, Gillanders KR, Stratford Bobbitt ME, Gascoigne EW, Leach SJ. Safety and efficacy of Gammaplex(R) in idiopathic thrombocytopenic purpura (ClinicalTrials.gov--NCT00504075). PLoS One. 2014 Jun 3;9(6):e96600. doi: 10.1371/journal.pone.0096600. eCollection 2014.

Helpful Links

• Sponsor website

Study record dates

Study Major Dates

• Study Start: September 1, 2007
• Primary Completion (Actual): August 1, 2011
• Study Completion (Actual): August 1, 2011

Study Registration Dates

• First Submitted: July 18, 2007
• First Submitted That Met QC Criteria: July 18, 2007
• First Posted (Estimate): July 19, 2007

Study Record Updates

• Last Update Posted (Estimate): March 1, 2013
• Last Update Submitted That Met QC Criteria: February 26, 2013
• Last Verified: February 1, 2013

More Information

Terms related to this study

• Keywords: Idiopathic Thrombocytopenic Purpura; Bleeding disorders; Immune System and Disorders
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Thrombocytopenia; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic; Physiological Effects of Drugs; Immunologic Factors; Antibodies; Immunoglobulins; Immunoglobulins, Intravenous; gamma-Globulins; Rho(D) Immune Globulin
• Other Study ID Numbers: GMX02